VX22-CFD-017 (NEMO)
J
James Chmiel
Primary Investigator
Not Recruiting
2 years - 5 years
All
Phase
N/A
20 participants needed
1 Location
Brief description of study
To evaluate the psychometric properties of the Preschool CFQ-R, including the ability of the Preschool CFQ-R to detect change and to define meaningful within patient change.
Detailed description of study
To assess patient and caregiver perceptions, as measured by PROs and CGROs, after treatment with TRIKAFTA compared with baseline.
To assess the clinical effectiveness of TRIKAFTA compared with baseline
measured by select clinical outcomes in a real-world setting.
To assess the clinical effectiveness of TRIKAFTA compared with baseline
measured by select clinical outcomes in a real-world setting.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: cystic fibrosis
-
Age: 2 years - 5 years
-
Gender: All
Inclusion Criteria:
Diagnosed with CF
Age 3-5
Exclusion Criteria:
Previously exposed to TRIKAFTA
Updated on
01 Aug 2024.
Study ID: 17634; PPUL-VERTEX-NEMO
